Why the study?
With prolonged survival, Duchenne muscular dystrophy patients progressively experience multisystem complications, prompting a need to comprehensively describe clinical status and propose a new scoring tool pooling heterogeneous functional domains.
Cohort (n=132)
No
A newly proposed multidisciplinary scoring tool effectively aggregates heterogeneous functional decline in Duchenne muscular dystrophy patients, highlighting significant deterioration from age 13 and the prognostic importance of cardiac function and steroid therapy.
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LoMauro et al. (2020) conducted a cohort in Duchenne muscular dystrophy (n=132). Global scoring tool was evaluated on Clinical status and functional decline across cardiac, respiratory, nutritional, ambulation, and scoliosis domains.
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